MOLECULAR MECHANISMS OF INHIBITOR DEVELOPMENT IN HEMOPHILIA
Mediterranean Journal of Hematology and Infectious Diseases, 2020 The development of neutralizing antibodies in hemophilia is a serious complication of factor replacement therapy. These antibodies, also known as “inhibitors”, significantly increase morbidity within the hemophilia population and lower the quality of ...
Davide Matino +2 more
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Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]
, 2002 The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Davidoff, AM +12 more
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Haematologica, 2016 Recent evidence suggests that patients with severe hemophilia B may have a less severe disease compared to severe hemophilia A. To investigate clinical, radiological, laboratory and histological differences in the arthropathy of severe hemophilia A and ...
Daniela Melchiorre +9 more
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Establishment of Care System for Hemophilia in China: Current Status and Future Prospect
罕见病研究, 2022 Hemophilia is a X-linked recessive hereditary bleeding disorders. The patients need to receive replacement treatment with coagulation factors in their whole lives.
YANG Renchi
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Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]
, 2001 Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A +5 more
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Experimental Biology and Medicine, 1931 Vines, 1 in 1920, while treating a patient with hemophilia observed that following the development of a local skin reaction to horse serum, not only did the bleeding cease, but there was also a reduction in the coagulation time of the capillary blood. In 1926, Mills 2 repeated the observations of Vines.
openaire +3 more sources
Spontaneous bleeding in a patient with malignant lymphoma: A case of acquired hemophilia [PDF]
, 2000 Background: Acquired hemophilia is a rare condition which can be associated with lymphoproliferative disease. Case Report: Eleven yea rs after the diagnosis of immunocytoma had been made, a 72-year-old man developed a high-titer factor VIII inhibitor. At
Brack, N. +5 more
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Porcine model of hemophilia A. [PDF]
PLoS ONE, 2012 Hemophilia A is a common X chromosome-linked genetic bleeding disorder caused by abnormalities in the coagulation factor VIII gene (F8). Hemophilia A patients suffer from a bleeding diathesis, such as life-threatening bleeding in the brain and harmful ...
Yuji Kashiwakura +15 more
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Endothelial cells derived from patients' induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A. [PDF]
, 2020 Hemophilia A (HA) is a bleeding disorder characterized by spontaneous and prolonged hemorrhage. The disease is caused by mutations in the coagulation factor 8 gene (F8) leading to factor VIII (FVIII) deficiency.
Agu, Emmanuel +9 more
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Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]
, 2003 A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM +4 more
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